Anemia Clinical Trial
— FORTISOfficial title:
Randomised, Open-label, Active-controlled, Multicentre, Comparative Study to Evaluate the Safety and Efficacy of Ferric Maltol (Iron (III)-Maltol Complex) (ST10) Oral Suspension Compared to Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, Incorporating a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years
The objective of the study is to compare the safety and gastrointestinal tolerability of ferric maltol oral suspension and ferrous sulfate oral liquid in children and adolescents aged 2 years to 17 years, and assess the safety and tolerability of ferric maltol oral suspension in children 1 month to less than 2 years, in the treatment of iron deficiency anaemia during the 12 weeks treatment period.
Status | Recruiting |
Enrollment | 98 |
Est. completion date | July 2024 |
Est. primary completion date | April 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month to 17 Years |
Eligibility | Inclusion Criteria: 1. Patient is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements. 2. Age =1 month and =17 years at the time of informed consent 3. Subjects must have iron deficiency anaemia defined by the following criteria, as measured by the central laboratory at the screening visit Haemoglobin thresholds define anaemia by age and gender: Children (1 m - < 5 yrs) <11.0 g/dl Children (5 yrs - < 12 yrs) <11.5 g/dl Children (12 yrs) <12.0 g/dl Female child (=13 yrs) <12.0 g/dl Male child (=13 yrs) <13.0 g/dl and Ferritin thresholds define anaemia by: ferritin <30 µg/L, or ferritin <50 µg/L with transferrin saturation (TSAT) <20%, 4. Female subjects of childbearing potential must agree to use a highly effective method of contraception (which includes complete abstinence) until study completion and for at least 4 weeks following their final study visit. Highly effective contraception is defined as a method which results in a low failure rate, i.e., less than 1% per year when used consistently and correctly, such as implants, injectables, some intrauterine contraceptive devices (IUDs), a vasectomised partner and oral contraceptive medications. The need for contraception and compliance with contraception requirements will be assessed at every visit for adolescent patients, and urine pregnancy testing will be performed at each visit for female subjects of childbearing potential. Exclusion Criteria: 1. Subject with anaemia due to any cause other than iron deficiency, including, but not limited to, a. Untreated or untreatable severe malabsorption syndrome 2. Subjects who have received prior to Screening: 1. Within 28 days intramuscular or intravenous (IV) injection or administration of depot iron preparation. 2. Within 7 days single agent iron preparations and during the study. 3. Within 12 weeks of blood transfusion or is scheduled to have blood transfusion or donation during the study period 4. Within 28 days erythropoiesis stimulating agents and during the study period 5. Within 14 days COVID-19 vaccination 3. Subjects with vitamin B12 or folic acid deficiency as determined by the central laboratory screening results. Subjects may start vitamin B12 or folate replacement and rescreen after at least 2 weeks. 4. Has concomitant disease that would significantly compromise iron absorption or absorbed iron utilization such as swallowing disorders and/or extensive small bowel resection. 5. History of active peptic ulcer 6. Has chronic renal disease (eGFR <60 mL/min/m2), as assessed at Screening based on serum creatinine. 7. Known hypersensitivity or allergy to either the active substance or excipients of ferric maltol or ferrous sulfate. 8. Has a known contraindication for treatment with iron preparations, e.g. haemochromatosis, chronic haemolytic disease, sideroblastic anaemia, thalassemia, or lead intoxication induced anaemia. 9. Impaired liver function as indicated by alanine aminotransferase (ALT) or aspartate transaminase (AST)>2.0 times upper normal limit as measured at the Screening visit. 10. Active acute inflammatory disease, including IBD flare or disease exacerbation, which in the opinion of the Investigator, is clinically significant. 11. Active chronic or acute infectious diseases requiring antibiotic treatment. 12. Pregnant or breast feeding. 13. Concomitant medical conditions with extensive active bleeding, other than menstrual cycles; subjects who suffer from menorrhagia may be included at the Investigator's discretion. 14. Scheduled or expected hospitalisation and/or surgery during the course of the study 15. Participation in any other interventional clinical study within 28 days prior to Screening. 16. Diagnosed to be COVID-19 positive by (SARS-CoV-2-RT-PCR positive) within 28 days prior to screening. 17. Cardiovascular, liver, renal, hematologic, psychiatric, neurologic, gastrointestinal, immunologic, endocrine, metabolic, respiratory or central nervous system disease that, in the opinion of the Investigator, may adversely affect the safety of the subject and/or objectives of the study drug or severely limit the lifespan of the subject. 18. Any other unspecified reason that, in the opinion of the Investigator or the Sponsor make the subject unsuitable for enrolment. |
Country | Name | City | State |
---|---|---|---|
Puerto Rico | BRCR Global Puerto | San Juan | |
United Kingdom | Noah's Ark Children's Hospital for Wales | Cardiff | |
United Kingdom | Royal Hospital for Sick Children - Edinburgh | Edinburgh | |
United Kingdom | Leicester Royal Infirmary | Leicester | |
United Kingdom | Alder Hey Children's NHS Foundation Trust | Liverpool | |
United Kingdom | Alder Hey Children's NHS Foundation Trust | Liverpool | |
United Kingdom | King's College Hospital | London | |
United Kingdom | Newham University Hospital | London | |
United Kingdom | Royal Manchester Children's Hospital | Manchester | |
United Kingdom | Nottingham University Hospitals | Nottingham | |
United States | Levine Cancer Institute | Charlotte | North Carolina |
United States | BRCR Global Texas | Edinburg | Texas |
United States | Penn State Hershey Children's Hospital | Hershey | Pennsylvania |
United States | Homestead Research Institute | Homestead | Florida |
United States | Clinical Research Prime | Idaho Falls | Idaho |
United States | Zion Research | Katy | Texas |
United States | Kissimmee Clinical Research Corp | Kissimmee | Florida |
United States | Sierra Clinical Research | Las Vegas | Nevada |
United States | Medical Research of Westcheste | Miami | Florida |
United States | Miami Clinical Research | Miami | Florida |
United States | Eminent Clinical Research and Associates | N. Lauderdale | Florida |
United States | Hasbro Children's Hospital | Providence | Rhode Island |
United States | Sun Research Institute | San Antonio | Texas |
United States | The Center for Clinical Trials | Saraland | Alabama |
United States | MultiCare Health System Institute for Research and Innovation | Tacoma | Washington |
Lead Sponsor | Collaborator |
---|---|
Shield Therapeutics |
United States, Puerto Rico, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Safety and gastrointestinal tolerability will be compared between ferric maltol oral suspension and ferrous sulfate oral liquid via summaries of treatment emergent adverse events (TEAEs), treatment emergent serious AEs (TESAEs) and treatment-emergent AEs | Safety and gastrointestinal tolerability will be compared between ferric maltol oral suspension and ferrous sulfate oral liquid via the incidence of treatment emergent adverse events (TEAEs), treatment emergent serious AEs (TESAEs) and treatment-emergent AEs (TEAEs) leading to premature discontinuation of study drug, estimated as the number of subjects with at least one event divided by the number of subjects in the safety population.
AEs will be categorised by primary system organ class and MedDRA preferred term as coded using the MedDRA dictionary. The number, intensity, relation to study medication and action taken will be described by incidence tables. SAEs will be discussed separately. |
12 weeks | |
Primary | Assess the effect on haemoglobin and iron markers in children and adolescents aged 1 month to 17 years after twice daily ferric maltol oral suspension administration for 12 weeks | 12 weeks | ||
Secondary | Assess the PK in children and adolescents aged 2 to 17 years | To assess the pharmacokinetics (PK) in children and adolescents aged 2 to 17 years after a single dose of ferric maltol oral suspension Visit 2 (PK Day 1), and after twice daily administration for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose, through measurement of serum iron, transferrin saturation (TSAT) and plasma maltol and maltol glucuronide | 12 weeks | |
Secondary | Assess the PK, in children aged 1 month to less than 2 years of age | To assess the PK, in children aged 1 month to less than 2 years of age after a single dose of ferric maltol oral suspension (Pre-assignment PK visit) and after twice daily administration for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose, through measurement of serum iron, transferrin saturation (TSAT), plasma and urine concentration of maltol and maltol glucuronide | 12 weeks | |
Secondary | Assess the effect, in children aged 1 month to less than 2 years of age | To assess the effect, in children aged 1 month to less than 2 years of age after a single dose of ferric maltol suspension (Pre-assignment PK visit), and after twice daily administration for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose, on serum transferrin, total and unsaturated iron binding capacity (TIBC, UIBC), ferritin | 12 weeks | |
Secondary | Assess the effect, in children aged 2 to 17 | To assess the effect, in children aged 2 to 17 after a single dose of ferric maltol suspension Visit 2 (PK Day 1), and after twice daily administration for at least 6 days, on Visit 3 (PK Day 2) after a single morning dose, on serum transferrin, total and unsaturated iron binding capacity (TIBC, UIBC), ferritin. | 12 weeks | |
Secondary | To compare the palatability from age-appropriate scoring system of ferric maltol oral suspension and ferrous sulfate oral liquid | 12 weeks |
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